efgartigimod alfa   

GtoPdb Ligand ID: 9777

Synonyms: ARGX-113
Compound class: Antibody
Comment: Efgartigimod alfa (ARGX-113) is a first-in-class antibody fragment designed for the treatment of patients with severe autoimmune diseases associated with high levels of pathogenic immunoglobulin G (IgG) autoantibodies [6]. It binds to the IgG receptor, major histocompatibility complex class I-like Fc receptor (FcRn, the protein product of the FCGRT gene). Efgartigimod alfa inactivates FcRn's IgG binding ability (the FcRn-IgG interaction normally protects IgG from degradation) thus facilitating accelerated catabolism of native pathogenic autoantibodies [3,5]. The mechanism of this drug action is discussed more comprehensively in Roopenian and Akilesh (2007) [4] and Vaccaro et al. (2005) [7].
Immunopharmacology Comments
Amelioration of experimentally induced MG in rats by FcRn blockade is reported by Liu et al. (2007) [2]. ARGX-113 has completed Phase 2 clinical trial for myasthenia gravis (MG). Although clinically meaningful and statistically significant benefit of ARGX-113 over placebo has been reported in press releases from the drug's developer ArgenX, no published results are available (as of Feb 2018).
Immunopharmacology Disease
Disease X-Refs Comment References
Myasthenia gravis Disease Ontology: DOID:437
OMIM: 254200
Orphanet: ORPHA589
Phase 2 clinical trial NCT02965573 in MG patients has been completed. FDA orphan drug for MG (granted Sept 2017) 1